Factors influencing the immediate post-DDAVP FVIII:C response
| . | n . | Basal FVIII:C, mean (min-max), IU/mL . | P value . | FVIII:C peak, mean (min-max), IU/mL . | P value . | FVIII:C recovery, mean (min-max) . | P value . |
|---|---|---|---|---|---|---|---|
| All | 361 | 0.34 (0.08-0.65) | 1.13 (0.19-2.69) | 2.85 (1.06-7.13) | |||
| F8 variants | |||||||
| Null∗ | 143 | 0.32 (0.08-0.65) | .0115 | 1.01 (0.19-2.26) | <.0001 | 2.85 (1.06-5.14) | .0038 |
| Non-null† | 218 | 0.35 [0.10-0.64] | 1.21 (0.44-2.69) | 3.15 (1.25-7.13) | |||
| Severity of the familial HA‡ | |||||||
| Severe/moderate | 216 | 0.32 (0.08-0.65) | .0049 | 1.04 (0.19-2.34) | <.0001 | 2.84 (1.06-6.10) | <.0001 |
| Mild | 145 | 0.35 (0.16-0.64) | 1.27 (0.44-2.69) | 3.31 (1.25-7.13) | |||
| Basal VWF:Ag§ | |||||||
| <0.70 | 127 | 0.32 (0.09-0.55) | .0183 | 1.21 (0.35-2.60) | .0047 | 3.32 (1.25-7.13) | <.0001 |
| ≥0.70 | 207 | 0.35 (0.08-0.65) | 1.08 (0.19-2.69) | 2.85 (1.06-5.64) | |||
| Blood type|| | |||||||
| O | 195 | 0.34 (0.09-0.65) | ns | 1.15 (0.19-2.69) | ns | 3.07 (1.06-6.95) | ns |
| Non-O | 118 | 0.34 (0.08-0.64) | .9259 | 1.15 (0.38-2.60) | .9511 | 2.99 (1.41-6.84) | .4342 |
| Age (y) | |||||||
| <18 | 125 | 0.32 (0.08-0.56) | .0156 | 1.10 (0.34-2.60) | ns | 2.95 (1.25-7.13) | ns |
| ≥18 | 236 | 0.35 (0.09-0.65) | 1.15 (0.19-2.69) | .2188 | 3.08 (1.06-6.95) | .2161 | |
| Body weight (kg) | |||||||
| <35 | 72 | 0.32 (0.08-0.56) | 1.01 (0.35-1.72) | 2.68 (1.25-4.41) | |||
| 35-70 | 271 | 0.34 (0.10-0.64) | ns | 1.15 (0.19-2.69) | .0105 | 3.12 (1.06-7.13) | .0007 |
| ≥70 | 18 | 0.41 (0.24-0.65) | .2015 | 1.36 (0.81-1.86) | 3.25 (1.80-6.10) | ||
| DDAVP doses¶ (μg/kg) | |||||||
| ≤0.3 | 263 | 0.34 (0.08-0.65) | ns | 1.11 (0.19-2.53) | ns | 2.94 (1.06-7.13) | .0005 |
| >0.3 | 83 | 0.32 (0.15-0.55) | .0795 | 1.21 (0.35-2.69) | .0625 | 3.36 (1.70-6.84) |
| . | n . | Basal FVIII:C, mean (min-max), IU/mL . | P value . | FVIII:C peak, mean (min-max), IU/mL . | P value . | FVIII:C recovery, mean (min-max) . | P value . |
|---|---|---|---|---|---|---|---|
| All | 361 | 0.34 (0.08-0.65) | 1.13 (0.19-2.69) | 2.85 (1.06-7.13) | |||
| F8 variants | |||||||
| Null∗ | 143 | 0.32 (0.08-0.65) | .0115 | 1.01 (0.19-2.26) | <.0001 | 2.85 (1.06-5.14) | .0038 |
| Non-null† | 218 | 0.35 [0.10-0.64] | 1.21 (0.44-2.69) | 3.15 (1.25-7.13) | |||
| Severity of the familial HA‡ | |||||||
| Severe/moderate | 216 | 0.32 (0.08-0.65) | .0049 | 1.04 (0.19-2.34) | <.0001 | 2.84 (1.06-6.10) | <.0001 |
| Mild | 145 | 0.35 (0.16-0.64) | 1.27 (0.44-2.69) | 3.31 (1.25-7.13) | |||
| Basal VWF:Ag§ | |||||||
| <0.70 | 127 | 0.32 (0.09-0.55) | .0183 | 1.21 (0.35-2.60) | .0047 | 3.32 (1.25-7.13) | <.0001 |
| ≥0.70 | 207 | 0.35 (0.08-0.65) | 1.08 (0.19-2.69) | 2.85 (1.06-5.64) | |||
| Blood type|| | |||||||
| O | 195 | 0.34 (0.09-0.65) | ns | 1.15 (0.19-2.69) | ns | 3.07 (1.06-6.95) | ns |
| Non-O | 118 | 0.34 (0.08-0.64) | .9259 | 1.15 (0.38-2.60) | .9511 | 2.99 (1.41-6.84) | .4342 |
| Age (y) | |||||||
| <18 | 125 | 0.32 (0.08-0.56) | .0156 | 1.10 (0.34-2.60) | ns | 2.95 (1.25-7.13) | ns |
| ≥18 | 236 | 0.35 (0.09-0.65) | 1.15 (0.19-2.69) | .2188 | 3.08 (1.06-6.95) | .2161 | |
| Body weight (kg) | |||||||
| <35 | 72 | 0.32 (0.08-0.56) | 1.01 (0.35-1.72) | 2.68 (1.25-4.41) | |||
| 35-70 | 271 | 0.34 (0.10-0.64) | ns | 1.15 (0.19-2.69) | .0105 | 3.12 (1.06-7.13) | .0007 |
| ≥70 | 18 | 0.41 (0.24-0.65) | .2015 | 1.36 (0.81-1.86) | 3.25 (1.80-6.10) | ||
| DDAVP doses¶ (μg/kg) | |||||||
| ≤0.3 | 263 | 0.34 (0.08-0.65) | ns | 1.11 (0.19-2.53) | ns | 2.94 (1.06-7.13) | .0005 |
| >0.3 | 83 | 0.32 (0.15-0.55) | .0795 | 1.21 (0.35-2.69) | .0625 | 3.36 (1.70-6.84) |
A monovariate analysis was performed to investigate the influence of different factors on the FVIII response to DDAVP, using the unpaired parametric t test to compare continuous variables between groups.
ns, nonsignificant.
Null variants include large deletions, intron 1 or 22 inversions, small nucleotide insertions/deletions causing a frameshift with a premature stop codon, and nonsense substitutions.
Non-null variants include missense variants, nucleotide substitutions in the promoter or splice sites, and small deletions of 10 to 14 nucleotides in intron 13.
Severity of the familial HA known in male relatives.
VWF:Ag levels were available for 334 of 361 carriers (93%).
ABO blood type data were available for 313 of 361 carriers (86%).
DDAVP doses were available for 346 of 361 carriers (96%).